Evidence map›Paper›PMID 42350385›Full record

ArticleNature communications2026

Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model.

Fang Wan, Jinchen He, Hong Ma, Debora PiresFerreira, Veena Kumanan, Ji Sun Lee, Xiupeng Chen, Ran He, Qin Su, Thomas L Gallagher and 9 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Nature communications, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

19 authors.

Fang WanHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
Jinchen HeHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
Hong MaViral Vector Core, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Debora PiresFerreiraHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.ORCID 0000-0001-7747-9599
Veena KumananHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
Ji Sun LeeHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
Xiupeng ChenHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.ORCID 0000-0001-5916-3953
Ran HeHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
Qin SuHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
Thomas L GallagherHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.ORCID 0000-0001-6381-6902
Sha ZhuNeushen Therapeutics US Inc., Burlington, MA, USA.
Gabriela Toro CabreraNeushen Therapeutics US Inc., Burlington, MA, USA.ORCID 0000-0002-8905-1589
Lingzhi ZhaoNeushen Therapeutics US Inc., Burlington, MA, USA.
Joan ShenNeushen Therapeutics US Inc., Burlington, MA, USA.
Alisha GruntmanHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
Robert H BrownDepartment of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, USA.ORCID 0000-0001-6062-1528
Zuoshang XuDepartment of Biochemistry & Molecular Biotechnology, University of Massachusetts Chan Medical School, Worcester, MA, USA. zuoshang.xu@umassmed.edu.ORCID 0000-0003-1209-334X
Guangping GaoHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA. guangping.gao@umassmed.edu.ORCID 0000-0003-0097-9012
Jun XieHorae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA. jun.xie@umassmed.edu.ORCID 0000-0001-9565-1567

Funding

Protein Arginine Deiminase 2 (PAD2) and Protein Citrullination in ALSR01NS118145 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI XU, ZUOSHANG · 2021 to 2025
$2.4M
NINDS NIH HHS R01 NS118145United States Department of Defense | United States Army | Army Medical Command | Congressionally Directed Medical Research Programs (CDMRP) AL240123
6 · The paper itself

Abstract

Adeno-associated virus (AAV)-mediated gene silencing offers a promising strategy for achieving durable therapeutic effects with a single administration. Mutations in the human superoxide dismutase 1 (hSOD1) gene, inherited in an autosomal dominant manner, lead to motor neuron degeneration in amyotrophic lateral sclerosis (ALS)-a fatal neurodegenerative disease with no effective treatment. In this study, we employed AAV9 to deliver to the SOD1

Indexed as

Amyotrophic Lateral SclerosisDependovirusGene SilencingGenetic TherapyGenetic VectorsSuperoxide Dismutase-1Administration, IntravenousAnimalsDisease Models, AnimalFemaleGene Therapy AgentsHumansMaleMiceMice, TransgenicMicroRNAsMicroRNAsSOD1 protein, humanSuperoxide Dismutase-1

Identifiers

PMID42350385
PMCPMC13303913

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.